uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington’s disease. Two patients are included so far, with one being given the therapy and the other being given an imitation surgery. After these two participants are monitored for 90 days, the trial will move forward.
About Huntington’s Disease
Huntington’s disease is a progressive, neurological condition that sees the deterioration of the brain. It causes the loss of mental and physical functions. This disease is very rare, affecting three to seven of every 100,000 people. It tends to affect those of European ancestry, with diagnosis typically occurring from age 30 to 40. The HTT gene is mutated in those with this condition, and it creates the instructions for the protein huntingtin. The exact purpose of this protein is unknown, but doctors believe that it is involved in the health of neurons. It accumulates in long chains due to the mutation and it will then bind to neurons which lose function and die. This gene is inherited in an autosomal dominant pattern, meaning only one parent has to pass down the mutated gene for a child to be affected.
Huntington’s symptoms fall into one of three categories: emotional issues, cognitive decline, or uncontrolled motor symptoms. The first form of symptoms may manifest as personality changes, mood swings, anxiety, depression, and impulsive behavior. Cognitive decline sees effects such as poor decision-making, forgetfulness, and issues with learning and retaining new information. The last category includes symptoms like twitching, shrugging, and issues with walking, coordination, and swallowing. Chorea, or uncontrolled movements, is one of the most common effects. Genetic testing is the major method used to diagnose this disease, as people are typically aware that Huntington’s runs in their family. Once a diagnosis is obtained and symptoms begin, treatment is symptomatic. Anti-psychotic drugs or dopamine-depleting drugs are used to suppress chorea. Doctors may also prescribe anti-depressants, neuroleptics, and antiepileptic drugs.
About the Study
This study will be randomized and double-blind, taking place in multiple centers across the United States. 26 patients will be given either AMT-130 or an imitation surgery in order to test the safety, efficacy, and tolerability of the gene therapy. The first 18 months will create the core study, while the next 42 months will be a follow-up.
Currently, only two patients have been treated. After 90 days, the Data Safety Monitoring Board (DSMB) will review the data and decide if the remaining participants can be dosed.
Huntington’s disease patients need viable treatments, so the success of this trial is vital to them. Hopefully, this phase and following ones will be successful.
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